Ignite Creation Date:
2024-05-05 @ 11:52 AM
Last Modification Date:
2024-10-26 @ 9:14 AM
Study NCT ID:
NCT00152113
Status:
COMPLETED
Last Update Posted:
2017-04-26
First Post:
2005-09-07
Brief Title:
Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Hematopoietic Stem Cell Transplantation for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Doppler Ultrasound Using Reduced-Intensity Conditioning and T-Cell Depleted HSC From Partially Matched Family Donors
Status:
COMPLETED
Status Verified Date:
2009-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Sickle cell disease is a life-long blood condition that can cause damage to the brain and other organs of the body Children may develop severe debilitating clinical states with stroke or abnormal blood flow to the brain Treatment generally includes chronic blood transfusions which may cause iron overload potentially leading to severe and sometimes fatal complications
Hematopoietic stem cell transplant using cells obtained from a sibling or an unrelated volunteer donor who is a perfect HLA match same tissue type for the recipient has shown to help and possibly cure sickle cell disease Unfortunately only about 10-20 of sickle cell patients have a HLA matched sibling donor and the likelihood of locating an appropriate HLA matched unrelated donor through the various donor registries is limited
Stem cells from partially HLA matched family members also called haploidentical transplant is an option currently being explored for this patient population This type of transplant has been used and found to be successful in some patients mostly those with cancers of the blood However there can be significant complications with haploidentical transplant primarily infection failure of the graft to grow graft failure and a disorder called graft-versus-host disease In addition few patients with sickle cell disease have undergone this procedure Therefore the risks and benefits of haploidentical transplants for patients with sickle cell disorder are not as well established as those using an HLA identical sibling or unrelated donor
The primary objective of this study is to assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy The treatment plan will be considered safe if there is not excessive toxicity Toxicity for this protocol is defined as graft failuregraft rejection severe acute GVHD or regimen related death within 100 days after the last cellular product administered
Of note the protocol was closed to accrual in September 2007 as we had met the stopping rules related to graft integrity graft failure and graft rejection Participants currently enrolled continue to be followed per protocol
Hematopoietic stem cell transplant using cells obtained from a sibling or an unrelated volunteer donor who is a perfect HLA match same tissue type for the recipient has shown to help and possibly cure sickle cell disease Unfortunately only about 10-20 of sickle cell patients have a HLA matched sibling donor and the likelihood of locating an appropriate HLA matched unrelated donor through the various donor registries is limited
Stem cells from partially HLA matched family members also called haploidentical transplant is an option currently being explored for this patient population This type of transplant has been used and found to be successful in some patients mostly those with cancers of the blood However there can be significant complications with haploidentical transplant primarily infection failure of the graft to grow graft failure and a disorder called graft-versus-host disease In addition few patients with sickle cell disease have undergone this procedure Therefore the risks and benefits of haploidentical transplants for patients with sickle cell disorder are not as well established as those using an HLA identical sibling or unrelated donor
The primary objective of this study is to assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy The treatment plan will be considered safe if there is not excessive toxicity Toxicity for this protocol is defined as graft failuregraft rejection severe acute GVHD or regimen related death within 100 days after the last cellular product administered
Of note the protocol was closed to accrual in September 2007 as we had met the stopping rules related to graft integrity graft failure and graft rejection Participants currently enrolled continue to be followed per protocol
Detailed Description:
Secondary objectives for this protocol include the following
To estimate 1-year overall and event free survival after transplantation An event is defined as toxicity graft failure death grade IIIIV acute GHVD or a sickle-related event stroke acute chest syndrome pain crisis
To obtain preliminary information regarding donor engraftment among different cell subsets including unsorted mononuclear cell and lymphoid fractions during the first year after transplant
To observe the rate of acute and chronic GVHD during the first year after transplant
To assess the proportion of research participants who experience poor graft integrity and therefore require additional donor stem cells or lymphocytes
To document the effect of stem cell transplant on the central nervous system as defined by radiological imaging and neuropsychological testing
To investigate immune reconstitution after transplantation
To estimate 1-year overall and event free survival after transplantation An event is defined as toxicity graft failure death grade IIIIV acute GHVD or a sickle-related event stroke acute chest syndrome pain crisis
To obtain preliminary information regarding donor engraftment among different cell subsets including unsorted mononuclear cell and lymphoid fractions during the first year after transplant
To observe the rate of acute and chronic GVHD during the first year after transplant
To assess the proportion of research participants who experience poor graft integrity and therefore require additional donor stem cells or lymphocytes
To document the effect of stem cell transplant on the central nervous system as defined by radiological imaging and neuropsychological testing
To investigate immune reconstitution after transplantation
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None